Supernus Pharmaceuticals, Inc.
Long Term Study of the Safety of OXC XR as in Pediatric Epilepsy Subjects
Conditions: Epilepsy
Follow-on study to continue evaluation of the safety of OXC XR as adjunctive therapy inpediatric epilepsy
SynCardia Systems. LLC
SynCardia Freedom Driver System Study
Conditions: Biventricular Failure
The purposes of this study are to confirm that the Freedom Driver System is a suitablepneumatic driver for clinically stable TAH-t subjects, and that patients and laycaregivers can be trained to manage the Freedom Driver System safely outside thehospital.
GE Healthcare
Expanded Access Protocol for [123I]mIBG for Patients With Known or Suspected Neuroblastoma
Conditions: Neuroblastoma
GE Healthcare has recently submitted a New Drug Application (NDA) for Iobenguane I 123Injection ([123I]mIBG (AdreView)) as a diagnostic nuclear imaging agent for the detectionof primary or metastatic neuroblastoma and pheochromocytoma. The present protocolestablishes an Expanded Access program to provide AdreView to pediatric medical centersand hospitals that treat neuroblastoma patients. AdreView will be provided for use indiagnostic assessment of patients with known or suspected neuroblastoma for whom there isan appropriate clinical indication for [123I]mIBG imaging.
Genzyme, a Sanofi Company
Alglucosidase Alfa Temporary Access Program
Conditions: Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiencyof a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA isused by the body's cells to break down glycogen (a stored form of sugar) withinspecialized structures called lysosomes. In patients with Pompe disease, an excessiveamount of glycogen accumulates and is stored in various tissues, especially heart andskeletal muscle, which prevents their normal function. The objective of this expandedaccess study is to provide patients with Pompe disease in the United States (US), accessto alglucosidase alfa produced from a scaled up manufacturing process for a limited timeuntil production at this scale is approved for commercial use by the Food and DrugAdministration.
Gilead Sciences
Aztreonam Lysine for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection
Conditions: Cystic Fibrosis, Pseudomonas Aeruginosa Airway Infection
The primary objective of this program is to provide expanded access to aztreonam lysinefor inhalation (AZLI) 75 mg prior to its commercial availability to patients with cysticfibrosis (CF) and chronic P. aeruginosa airway infection who have limited treatmentoptions and are at risk for disease progression.
BioMarin Pharmaceutical
Sapropterin Expanded Access Program
Conditions: Phenylketonuria
The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due toPhenylketonuria (PKU) access to sapropterin dihydrochloride and to collect moreinformation about the safety of the drug in an expanded access program (EAP) untilcommercial product is available.
Alexion Pharmaceuticals, Inc.
The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol
Conditions: Hemoglobinuria, Paroxysmal
The primary objective is to provide access to eculizumab for PNH patient pendingcommercial availability.
ApoPharma
Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease
Conditions: Iron Overload
Patients who have iron overload due to chronic blood transfusions and have developedheart failure or who are at high risk of heart failure because of the high levels of ironin their hearts, will be treated with deferiprone, an investigational drug, incombination with deferoxamine (Desferal). Some studies suggest that deferiprone may bebetter than deferoxamine in removing iron from the heart and improving heart function,and that using both drugs together may remove more iron. Participants would make a clinicvisit for lab studies each week, and would continue to take deferiprone for as long astheir physician feels it is useful in their care.
Tibotec Pharmaceuticals, Ireland
TMC114-C226: An Early Access Program to Evaluate the Long-term Safety and Tolerability of TMC114 Combined With a Low Dose of Ritonavir (TMC114/r) With Other Antiretrovirals, for HIV-1 Infected Patients Who Have Failed Multiple Antiretroviral Regimens.
Conditions: HIV Infections
The purpose of this study is to provide early access to TMC114 (a protease inhibitor) forHIV-1 infected patients with limited or no treatment options, who have failed multipleantiretroviral (ARV) regimens, and to evaluate the longer-term safety and tolerability ofTMC114/r in combination with other antiretrovirals
AIM ImmunoTech Inc.
Ampligen in Chronic Fatigue Syndrome
Conditions: Chronic Fatigue Syndrome
This is an open label study of Ampligen in patients with chronic fatigue syndrome.